SerpinPC in Hemophilia

Pursuing a potential best-in-class treatment for hemophilia

Hemophilia is a rare genetic bleeding disorder caused by a deficiency in clotting factor activity, leading to spontaneous and persistent bleeding. Currently, there is no subcutaneous treatment option for hemophilia B in the US.

Our team is developing SerpinPC, a novel potential subcutaneous treatment for hemophilia B patients, who today lack a convenient, subcutaneous, safe and effective treatment option, with potential expansion into hemophilia A and other rare bleeding disorders.

Drug Candidate

SerpinPC is an investigational, subcutaneously delivered biologic of the serpin family of proteins, designed to allow more thrombin to be generated by inhibiting activated protein C (APC). Clinical data to date has shown that treatment with SerpinPC results in a significant reduction in bleeding, with a favorable safety and tolerability profile, including no observed thrombosis.¹

References: 1. Baglin T, Koch A, Mocanu I, Makhaldiani L, Huntington JA. SerpinPC in persons with severe hemophilia (PwH): updated results from a multi-center, multi-part, first-in-human study. Blood. 2022;140(Supplement 1): 460-461.

Novel MOA

Ongoing Clinical Studies

SerpinPC is an investigational agent that has not been approved by the FDA or any other regulatory authority.

SerpinPC is currently in registrational studies for the treatment of hemophilia B, with or without inhibitors.

Registrational studies:

PRESent-2 (NCT05789524): SerpinPC in moderately severe to severe hemophilia B without inhibitors, and severe hemophilia A with and without inhibitors

PRESent-3 (NCT05789537): SerpinPC in hemophilia B with inhibitors

PRESent-5 (NCT05605678): A global, non-interventional study to prospectively evaluate bleeding episodes and treatment use in participants with hemophilia

Other ongoing studies:

AP-0101 (NCT04073498): The safety and tolerability of SerpinPC in healthy men and in men with severe blood disorders (hemophilia A and B)

For additional detail, please visit www.clinicaltrials.gov (US), www.clinicaltrialsregister.eu (EU), or registries in other jurisdictions.

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